Gene therapy VG901 tested in people with retinitis pigmentosa from CNGA1 mutations
Tests a gene therapy injected into the eye in adults with inherited retinitis pigmentosa.
Plain-language title generated from the registry record
Registry title: Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
Key facts
- Where
- Munich and Tuebingen, GermanySee the 2 sites
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 64 years old?
Source: NCT06291935, who can take part
Where in Germany
- Ludwig-Maximilians-Universitaet MuenchenMunich, GermanyTaking people
- Universitaetsklinikum Tuebingen AöRTuebingen, GermanyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- VeonGen Therapeutics GmbH
- Trial code
- NCT06291935EU 2023-504383-42-00
- Record updated
- 28 March 2025
- Original description
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa. The main questions the study aims to answer are:Read full original descriptionShow less
A Prospective, Open-label, Phase 1b, Single-arm, Safety Study of an Intravitreal Application of a Recombinant Adeno-associated Virus Vector Expressing CNGA1 (AAV2.NN-CNGA1) in Patients With Retinitis Pigmentosa Due to CNGA1 Mutations
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa. The main questions the study aims to answer are:
* What is the best tolerated dose and are there any side effects, in particular any inflammatory reactions post drug administration? * Are there any early signs of efficacy on visual function?
Participants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the Protocol.
Inclusion: To be eligible for study entry, subjects must satisfy all the following criteria:; Able to understand and willing to consent to study participation by a written informed consent; Male or female ≥ 18 years of age; Clinical diagnosis of RP; Confirmed pathogenic, biallelic variants in the CNGA1 gene; Ellipsoid zone (EZ) length of the fovea of ≥ 3000 μm in the study eye.
Exclusion: Ocular surgery, intravitreal or subretinal implantation of a medical device (within 6 months of screening); Any other retinopathy due to other diseases - including, but not limited to arterial hypertension, previous vascular retinal occlusion, trauma or acquired inflammatory diseases, contraindication to pharmacological mydriasis (e.g., history of angle block glaucoma), diabetes (diabetic retinopathy including macular oedema); Individuals performing any other therapy for RP within 3 months before the study, such as - but not limited to - transcorneal electrostimulation; Current, or recent, participation in other study/ or administration of investigational biologic agent within 3 months of Screening; Use of any investigational agent, or systemic corticosteroids, or other immunosuppressive drug(s) within 3 months before Screening; Previous receipt of any AAV gene therapy product; Additional interfering ocular conditions which would impact study results (e.g., ocular opacity and advanced cataract, uveitis, amblyopia); Mutations known to cause inherited retinal disease other than biallelic variants in the CNGA1 gene; History of ocular infection with herpes simplex virus; History of ocular malignancies; History of disorders of the internal retina (e.g., retinal detachment); Systemic conditions (e.g., autoimmune disorders) which may affect study participation or outcome measures; History of immunodeficiency or other medical conditions which may increase the risk of VG901 administration; History of allergy or sensitivity to any compound used in the study; Any condition which leads the investigator to believe that subject cannot comply with the protocol requirements or that may place the subject at an unacceptable risk from participating; Patients with uncontrolled diabetes (HbA1c > 7%); Systemic illness (e.g., hepatitis or human immunodeficiency virus [HIV] infection) or medically relevant abnormal laboratory values (3 x upper limit of normal [ULN]) in blood analysis including renal and hepatic function; Subjects with increased risk of bleeding (i.e., use of anticoagulants or anti-platelet agents within 7 days before VG901 administration and subjects with international normalized ratio > 2 or Quick < 50% or partial thromboplastin time > 50 seconds, thrombocytopenia, as well as any other known coagulopathy); Subjects will be excluded from the study if one or more of the following statements are applicable to either eye:; History or presence of glaucoma; Absence of visual function on the contralateral eye; Any damage to the optic nerve; Contraindications to systemic immunosuppression; Subject/partner of childbearing potential unwilling to use adequate contraception for the period between Screening and 30 days after treatment, defined as the period from Screening until 30 days after treatment (defined as administration of therapeutic to the eye); For females of childbearing potential, a positive pregnancy test at Screening or Baseline; Females who are breastfeeding.